Red blood cells

The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for GlaxoSmithKline’s (GSK) Promacta/Revolade (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anaemia (SAA) who have had insufficient response to immunosuppressive therapy.

The FDA breakthrough therapy designation was based on the results from an open-label, Phase II National Institute of Health (NIH) study (09-H-0154) of eltrombopag conducted in 43 heavily pre-treated SAA patients with an insufficient response to IST.

The breakthrough therapy designation includes all the features of the fast track designation, as well as more intensive guidance from the FDA on a drug’s clinical development programme.

FDA breakthrough therapy status is aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.

The company said that eltrombopag is not approved or licensed anywhere in the world for use in this rare disorder SAA.

"FDA breakthrough therapy status is aimed at accelerating the development and review times of drugs for serious or life-threatening conditions."

Eltrombopag olamine is a small-molecule, nonpeptide thrombopoietin receptor agonist with megakaryopoiesis-stimulating activity.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

It is currently under development for the treatment of thrombocytopenia with chronic hepatitis C and compensated liver cirrhosis, haeme-oncology-related thrombocytopaenia, acute myelocytic leukaemia, myelodysplastic syndrome and aplastic anaemia.

In SAA, the bone marrow fails to make enough new blood cells, and currently there are no approved therapies available for SAA patients unresponsive to initial immunosuppressive therapy (IST).

Out of those patients unresponsive to initial IST, about 40% die from infection or bleeding within five years of their diagnosis.


Image: Eltrombopag is marketed under the brand name Revolade in Europe. Photo: courtesy of freedigitalphotos.net.